Recode Therapeutics, Inc.

Brandon A Wustman

Mirko Hennig

David J Lockhart

Daniella Ishimaru

Yufeng Wang

Sakya Sing Mohapatra

Vladimir Grigor'evich Kharitonov

Ali Ahmed Alfaifi

Jackson Eby

Julia Jung-un Baek

Shuang Li

Joseph S Cefalu

Vladimir Kharitonov

Rumpa Bhattacharjee

Michael Torres

Dmitri Boudko

Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for primary ciliary dyskinesia (PCD), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject.

Lipid nanoparticle compositions and uses thereof

Compositions of polynucleotide(s), pharmaceutical compositions thereof, and methods of use thereof are disclosed. A polynucleotide may encode for a cystic fibrosis transmembrane conductance regulator (CFTR) protein or a functional fragment thereof. The polynucleotide may be assembled with a lipid composition for targeted delivery to a cell or an organ, such as a lung cell or a lung of a subject. Methods for enhancing an expression or activity of CFTR protein in a cell are provided. Methods for treating a subject having or suspected of having a CFTR-associated condition are also provided.

Polynucleotide compositions, related formulations, and methods of use thereof

Described herein are compositions, kits, and methods for potent delivery to a cell of a subject. The cell can be of a particular cell type, such as a basal cell, a ciliated cell, or a secretory cell. In some cases, the cell can be a lung cell of a particular cell type. Also described herein are pharmaceutical compositions comprising a therapeutic or prophylactic agent assembled to a lipid composition. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Further described herein are high-potency dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.

Compositions and methods for targeted delivery to cells

Growing community of inventors

San Diego, CA, United States of America

Brandon A Wustman